Pa­tient-fo­cused drug de­vel­op­ment: New FDA draft guid­ance delves in­to what’s im­por­tant

The FDA on Mon­day pub­lished the sec­ond in a se­ries of four guid­ance doc­u­ments on pa­tient-fo­cused drug de­vel­op­ment, with this lat­est draft help­ing spon­sors iden­ti­fy what is most im­por­tant to pa­tients with re­spect to their ex­pe­ri­ence with dis­ease bur­den and treat­ment.

In de­ter­min­ing what’s im­por­tant, the FDA rec­om­mends that spon­sors use qual­i­ta­tive re­search meth­ods, which can gen­er­ate in-depth thoughts from pa­tients in their own words through in­ter­views or fo­cus groups, quan­ti­ta­tive re­search meth­ods, which can use sta­tis­ti­cal meth­ods to sum­ma­rize the col­lec­tive pa­tient ex­pe­ri­ence da­ta, or mixed-meth­ods re­search, which can com­bine qual­i­ta­tive and quan­ti­ta­tive meth­ods to un­der­stand the pa­tient ex­pe­ri­ence.

The 45-page draft dis­cuss­es sev­er­al ways to col­lect qual­i­ta­tive in­for­ma­tion, in­clud­ing via one-on-one in­ter­views and fo­cus groups, with hints on how to ask the right types of ques­tions.

On quan­ti­ta­tive meth­ods, the draft ex­plains dif­fer­ent sur­veys and what to con­sid­er, in­clud­ing ex­am­ples of what could po­ten­tial­ly be lead­ing or dou­ble-neg­a­tive ques­tions.

Ex­am­ples of mixed-method study de­signs are al­so ex­plained fur­ther, along­side ques­tions re­searchers should ask them­selves and rea­sons to use a mixed-method de­sign.

In ad­di­tion, the draft in­cludes a sec­tion on spe­cif­ic pop­u­la­tions, cul­tur­al­ly di­verse pop­u­la­tions and con­sid­er­a­tions for us­ing so­cial me­dia, which dis­cuss­es the im­por­tance of keep­ing the var­i­ous so­cial plat­forms and users in mind.

“When pos­si­ble, so­cial me­dia re­search should ex­am­ine a va­ri­ety of so­cial me­dia net­works and com­mu­ni­ties to ob­tain da­ta that can be most gen­er­al­ized to the pop­u­la­tion of in­ter­est,” the agency adds. The draft al­so in­cludes six ap­pen­dices, in­clud­ing a glos­sary of terms and dif­fer­ent types of ex­am­ples for each of the re­search meth­ods.

The re­lease of the draft guid­ance co­in­cides with the Mon­day re­lease of a new per­spec­tive ar­ti­cle on en­gag­ing pa­tients co-au­thored by both FDA and EMA of­fi­cials in Na­ture Re­views Drug Dis­cov­ery.

The per­spec­tive fo­cus­es on ini­tia­tives from both reg­u­la­tors that have al­lowed for fur­ther pa­tient or pub­lic en­gage­ment, and fur­ther en­gage­ment be­tween them.

“Our re­spon­si­bil­i­ty is the same for all pa­tients: to en­sure that they re­ceive the safest, most ef­fec­tive med­i­cines and that, as much as pos­si­ble, their needs and con­cerns are con­sid­ered in the eval­u­a­tion process,” EMA and FDA of­fi­cials wrote. “Giv­en the glob­al as­pects of med­i­cine reg­u­la­tion, col­lab­o­rat­ing and shar­ing ex­pe­ri­ences, in­for­ma­tion and re­sources be­tween agen­cies is al­so vi­tal, and ex­changes such as those seen through the col­lab­o­ra­tion be­tween the EMA and the FDA are help­ing to im­prove pa­tients’ trust in and un­der­stand­ing of the reg­u­la­to­ry process.”

The FDA, mean­while, which pre­vi­ous­ly re­leased the first pa­tient-fo­cused drug de­vel­op­ment draft guid­ance in June 2018, un­der this new ini­tia­tive cre­at­ed by the 21st Cen­tu­ry Cures Act, will al­so re­lease two oth­er guid­ance doc­u­ments deal­ing with ap­proach­es to iden­ti­fy and de­vel­op meth­ods to mea­sure im­pacts in clin­i­cal tri­als and meth­ods, stan­dards and tech­nolo­gies to col­lect and an­a­lyze clin­i­cal out­come as­sess­ment da­ta for reg­u­la­to­ry de­ci­sion-mak­ing.

Pa­tient-Fo­cused Drug De­vel­op­ment: Meth­ods to Iden­ti­fy What Is Im­por­tant to Pa­tients Guid­ance for In­dus­try, Food and Drug Ad­min­is­tra­tion Staff, and Oth­er Stake­hold­ers


RAPS: First pub­lished in Reg­u­la­to­ry Fo­cus™ by the Reg­u­la­to­ry Af­fairs Pro­fes­sion­als So­ci­ety, the largest glob­al or­ga­ni­za­tion of and for those in­volved with the reg­u­la­tion of health­care prod­ucts. Click here for more in­for­ma­tion.

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

Vas Narasimhan (AP Images)

UP­DAT­ED: Still held down by clin­i­cal hold, No­var­tis' Zol­gens­ma falls fur­ther be­hind Bio­gen and Roche as FDA asks for a new piv­otal study

Last October, the FDA slowed down Novartis’ quest to extend its gene therapy to older spinal muscular atrophy patients by slapping a partial hold on intrathecal administration. Almost a year later, the hold is still there, and regulators are adding another hurdle required for regulatory submission: a new pivotal confirmatory study.

The new requirement — which departs significantly from Novartis’ prior expectations — will likely stretch the path to registration beyond 2021, when analysts were expecting a BLA submission. That could mean more time for Biogen to reap Spinraza revenues and Roche to ramp up sales of Evrysdi in the absence of a rival.

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David Berry (Flagship)

Flag­ship's next big tech­no­log­i­cal bet? The cloud

Earlier this month, Flagship announced their big bet on the software half the industry is talking about, launching the AI and machine learning startup. Now, they and a couple other investors are gambling $100 million on a software that much of the public generally thinks of as a cool, IT afterthought: cloud computing.

The idea, says founder and Flagship partner David Berry, is one of scale: The sheer magnitude of biological data that you can store on cloud technology is unprecedented. And that size, when leveraged properly, can allow you to ask questions and form insights that are similarly unprecedented.

FDA commissioner Stephen Hahn at the White House (AP Images)

Un­der fire, FDA to is­sue stricter guid­ance for Covid-19 vac­cine EUA this week — re­port

The FDA has been insisting for months that a Covid-19 vaccine had to be at least 50% effective – a measure of transparency meant to shore public trust in the agency and in a vaccine that had been brought forward at record speed and record political pressure. But now, with concerns of a Trump-driven authorization arriving before the election, the agency may be raising the bar.

The FDA is set to release new guidance that would raise safety and efficacy requirements for a vaccine EUA above earlier guidance and above the criteria used for convalescent plasma or hydroxychloroquine, The Washington Post reported. Experts say this significantly lowers the odds of an approval before the election on November 3, which Trump has promised despite vocal concerns from public health officials, and could help shore up public trust in the agency and any eventual vaccine.

Rep. Andy Harris (R-MD) (Tasos Katopodis/Pool via AP Images)

The mi­cro-cap that tapped a mask-skep­tic con­gress­man for their Covid DSMB is ap­ply­ing for an EUA. Their ev­i­dence? 21 pa­tients

NeuroRx, the tiny biotech that came under fire last week after Politico reported they selected a congressman and two other acquaintances of the CEO to supervise their Covid-19 drug trial, announced today that they will ask the FDA to authorize their drug based on the results of just 21 patients.

Such an application would test the agency’s standards of evidence for an EUA, which have already come under scrutiny after controversial authorizations for convalescent plasma and hydroxychloroquine. The only other company to discuss their intention to file for an EUA, Eli Lilly, did so after results came back from a randomized control study testing their antibody in over 450 patients.

PhII Alzheimer's fail­ure deals new blow to Roche, AC Im­mune — but the tau hy­poth­e­sis is far from dead

The leading anti-tau antibody has failed its first Phase II testing, casting a shadow on a popular target (just trailing amyloid beta) for Alzheimer’s disease.

Roche and AC Immune are quick to acknowledge disappointment in the topline readout, which suggested that semorinemab did not reduce cognitive decline among patients with early Alzheimer’s disease, who are either just starting to have symptoms or have mild manifestations.

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Covid-19 roundup: J&J be­gins piv­otal Phase III tri­al for vac­cine; Con­tro­ver­sial hu­man chal­lenge tri­als to be­gin in Lon­don — re­port

Johnson & Johnson announced it’s beginning a pivotal Phase III trial for its Covid-19 candidate, JNJ-78436735 — the first single-dose vaccine in this stage.

The Phase III trial, dubbed ENSEMBLE, will enroll 60,000 patients worldwide, making it the largest Phase III study of a Covid-19 vaccine to date. J&J said the candidate achieved positive interim results in a Phase I/IIa study, which will be published “imminently.” There’s a possibility that the first batches will be ready for potential emergency use in early 2021, according to the company.

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Patrick Enright, Longitude co-founder (Longitude)

As its biotechs hit the pan­dem­ic ex­it, Lon­gi­tude rais­es $585M for new neu­ro, can­cer, ag­ing and or­phan-fo­cused fund

The years have been kind to Longitude Capital. This year, too.

A 2006 spinout of Pequot Capital, its founders started their new firm just four years before the parent company would go under amid insider trading allegations. Their first life sciences fund raised $325 million amid the financial crisis, they added a second for $385 million and then in, 2016, a third for $525 million. In the last few months, the pandemic biotech IPO boom netted several high-value exits from those funds, as Checkmate, Vaxcyte, Inozyme and Poseida all went public.

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Scoop: ARCH’s Bob Nelsen is back­ing an mR­NA up­start that promis­es to up­end the en­tire man­u­fac­tur­ing side of the glob­al busi­ness

For the past 2 years, serial entrepreneur Igor Khandros relied on a small network of friends and close insiders to supply the first millions he needed to fund a secretive project to master a new approach to manufacturing mRNA therapies.

Right now, he says, he has a working “GMP-in-a-box” prototype for a new company he’s building — after launching 3 public companies — which plans to spread this contained, precise manufacturing tech around the world with a set of partners. He’s raised $60 million, recruited some prominent experts. And not coincidentally, he’s going semi-public with this just as a small group of pioneers appears to be on the threshold of ushering in the world’s first mRNA vaccines to fight a worldwide pandemic.

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